The Lancet Publishes CSL Behring’s Alpha-1 Antitrypsin Deficiency Study Data

Important New Findings Published in The Lancet Support the Use of Zemaira® [Alpha1-Proteinase Inhibitor (Human)] in Patients with Alpha-1 Antitrypsin Deficiency (AATD)

Marburg, Germany — 01 June 2015

Findings from CSL Behring’s RAPID study, the largest placebo-controlled trial ever conducted in patients with alpha-1 antitrypsin deficiency (AATD), demonstrate that the use of Alpha1-Proteinase Inhibitor therapy may slow the progressive loss of lung tissue experienced by these critically ill patients. According to findings of RAPID (Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency), published by The Lancet, patients with AATD treated with Alpha1-Proteinase Inhibitor therapy exhibited a lower annual rate of lung density decline compared to placebo, when measured using chest computed tomography, at full inspiration.

“RAPID is regarded as a landmark study validating almost two decades of focus on the lung-density endpoint as the most sensitive way to track lung tissue decline and the seven-year collaboration of an international team of investigators,” said Kenneth R. Chapman, MD, Director of the Asthma & Airway Centre at the University Health Network in Toronto, and lead author of the paper. “Our findings provide additional evidence that treatment with an Alpha1-Proteinase Inhibitor may slow the accelerated loss of lung tissue that is a characteristic of this potentially debilitating disease.”

AATD is a hereditary condition that can severely affect a patient’s lung function. The condition is marked by a low level or absence of alpha-1-proteinase inhibitor (A1-PI), a natural protein that inhibits neutrophil elastase, thereby preventing destruction of lung tissue. Severe deficiency of A1-PI is associated with a strong tendency for the development of emphysema, a form of chronic obstructive pulmonary disease (COPD), and can significantly impact everyday life and life expectancy. According to a recent registry, emphysema affects 54 percent of diagnosed Alpha-1-deficient patients.

On average, it takes over seven years from the time a patient’s lung symptoms first appear until a proper diagnosis is made. And nearly half (43 percent) of patients see at least three physicians before being diagnosed. Approximately 90 percent of individuals with AATD are not diagnosed or are thought to have other conditions, such as asthma or smoking-related COPD.

"We are excited that the results of this important study in Alpha-1 have been published in the highly-respected journal The Lancet,” said John Walsh, co-founder, President and CEO of the Alpha-1 Foundation. “We commend CSL Behring for their outstanding commitment to the Alpha-1 community and advancing the understanding and treatment of the disease. These results further support the use of augmentation therapy in the treatment of Alpha-1, and we hope they bolster efforts of Alpha-1 communities around the world to win access to therapy.”

Study Design and Findings

According to study protocol, the effect of CSL Behring’s Alpha1-Proteinase Inhibitor therapy on the progression of emphysema, the primary endpoint of the study, was measured by computed tomography scan over 24 months and was assessed by the annual rate of lung density loss at total lung capacity (TLC) and functional residual capacity (FRC) combined. The annual rate of lung density loss at TLC and FRC combined were not statically different in treatment group compared to placebo group. However, results showed a 34 percent reduction in the annual rate lung density decline compared with placebo when measured at TLC (or full inspiration).

Serious adverse events were not statistically different between groups with one death in the A1-PI group and three deaths in the placebo group.

RAPID was a multicenter, double-blind, randomized, parallel group, placebo-controlled study comparing the efficacy and safety of CSL Behring’s Alpha1-Proteinase Inhibitor therapy with placebo in patients with emphysema due to AATD. The participants, 180 non-smokers aged 18-65, were randomly assigned to receive A1-PI intravenously 60 mg/kg weekly or placebo.

About Zemaira

Zemaira is a highly-purified form of Alpha1 Proteinase Inhibitor (human) currently approved in Brazil, New Zealand, and the US, where it is indicated for chronic augmentation and maintenance therapy in adults with Alpha1 deficiency and clinical evidence of emphysema.

CSL Behring is seeking additional approvals around the world for this A1-PI therapy, which is currently under review with the European Medicines Agency.

Zemaira is contraindicated in patients with a history of severe systemic reaction to the product or to A1-PI protein, including anaphylaxis. Due to the risk of severe hypersensitivity, Zemaira is also contraindicated in immunoglobulin A-deficient patients with antibodies against IgA.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit



Jennifer Seiler
CSL Behring
O: 610-878-4802